Biopharmconsortium Blog

Expert commentary from Haberman Associates biotechnology and pharmaceutical consulting.

Posts filed under: Rare diseases

Strimvelis (GSK2696273), a gene therapy for a deadly immunodeficiency in children, expected to reach the European market in mid-2016

TweetOur recent book-length report, Gene Therapy: Moving Toward Commercialization was published by Cambridge Healthtech Institute in November 2015. As indicated by its title, the report focuses on clinical-stage gene therapy programs that are aimed at commercialization, and the companies that are carrying out these programs. Until recently, gene therapy was thought of as a scientifically-premature…

Gene therapy for hemophilia—an update

Tweet  The boy pictured above is Tsarevich Alexei Nikolaevich of Russia, who lived between 1904 and 1918, and was the heir to the throne of Imperial Russia. He is arguably the most famous hemophiliac in history. Alexei suffered from hemophilia B, a form of hemophilia that was passed from Queen Victoria of Britain through two…

Can adoptive cellular immunotherapy successfully treat metastatic gastrointestinal cancers?

Tweet  On September 6, 2014, we published an article on this blog announcing the publication of our book-length report, Cancer Immunotherapy: Immune Checkpoint Inhibitors, Cancer Vaccines, and Adoptive T-cell Therapies, by Cambridge Healthtech Institute (CHI). In that article, we cited the example of the case of a woman with metastatic cholangiocarcinoma (bile-duct cancer), which typically…

Spark Therapeutics’ retinal disease gene therapy SPK-RPE65 may reach the U.S. market in 2017

Tweet  As we discussed in our November 16, 2015 article on this blog, Spark Therapeutics (Philadelphia, PA) recently announced positive top-line results from the Phase 3 pivotal trial of SPK-RPE65, a gene therapy for treatment of inherited retinal diseases (IRDs) caused by mutations in the gene for RPE65.  At a later scientific meeting, the company…

“Our Promise to Nicholas”, Batten disease, and gene therapy

Tweet  Russell’s Garden Center, on Route 20, a family-owned business established in 1876, is a unique Wayland MA institution. When you shop at Russell’s and approach the check-out counter with your plants, flowers, or other purchases, you will see a donation box for a rare-disease charity called “Our Promise to Nicholas Foundation”. This charity is…

Gene Therapy Report Published By CHI Insight Pharma Reports

Tweet  On November 6, 2015, Cambridge Healthtech Institute (CHI) announced the publication of a new book-length report, Gene Therapy: Moving Toward Commercialization, by Allan B. Haberman, Ph.D. As demonstrated by several late-breaking news items that appeared as our report was in the process of publication, gene therapy is a “hot”, fast-moving field. For example: On…

Vertex cystic fibrosis therapeutics update

Tweet As we said in our September 10, 2014 article, we intended to post updates on companies that we had been following on our blog, and that have achieved significant progress in recent months. So far, we have covered Agios and Zafgen. Both of these companies were featured in Boston-area meetings in October—Zafgen in Xconomy…

Obesity therapeutics update

TweetThe Biopharmconsortium Blog has over the years included numerous articles about obesity, and the attempts of researchers and companies to develop treatments for this disease. Obesity, which has historically been considered the result of “lack of willpower” or other behavioral issues, was recognized as a disease by the American Medical Association in June 2013. This…

RNAi therapeutics stage a comeback

TweetNot so long ago, the once-promising field of RNA interference (RNAi)-based drugs was on the downswing. This was documented in our August 22, 2011 article on this blog, entitled “The Big Pharma Retreat From RNAi Therapeutics Continues”. That article discussed the retreat from RNAi drugs by such Big Pharma companies as Merck, Roche, and Pfizer….

Agios Pharmaceuticals becomes a clinical-stage company!

TweetIn a news release on September 23, 2013, Agios Pharmaceuticals (Cambridge, MA) announced that it had initiated its first clinical study. The company further discussed its early clinical and preclinical programs in its press release on its Third Quarter financial report, dated November 7, 2013. Specifically, the company initiated a Phase 1 muticenter clinical trial…